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Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy

Primary Purpose

Mitochondrial Myopathy

Status
Completed
Phase
Not Applicable
Locations
Study Type
Interventional
Intervention
thioctic acid
Sponsored by
National Center for Research Resources (NCRR)
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Mitochondrial Myopathy focused on measuring inborn errors of metabolism, mitochondrial myopathy, rare disease

Eligibility Criteria

0 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Long standing ophthalmoparesis and fatiguable weakness of the limbs Mild ataxia and no central nervous system involvement History of mitochondrial DNA deletion and a measurable biochemical defect of the respiratory chain Steady deterioration in skeletal muscle mass and power over 5 years --Prior/Concurrent Therapy-- Previous participation in studies of muscle disease natural history (CRC Protocol 183A)

Sites / Locations

    Outcomes

    Primary Outcome Measures

    Secondary Outcome Measures

    Full Information

    First Posted
    February 24, 2000
    Last Updated
    June 23, 2005
    Sponsor
    National Center for Research Resources (NCRR)
    Collaborators
    University of Rochester
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    1. Study Identification

    Unique Protocol Identification Number
    NCT00004770
    Brief Title
    Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy
    Study Type
    Interventional

    2. Study Status

    Record Verification Date
    December 2001
    Overall Recruitment Status
    Completed
    Study Start Date
    October 1995 (undefined)
    Primary Completion Date
    undefined (undefined)
    Study Completion Date
    undefined (undefined)

    3. Sponsor/Collaborators

    Name of the Sponsor
    National Center for Research Resources (NCRR)
    Collaborators
    University of Rochester

    4. Oversight

    5. Study Description

    Brief Summary
    OBJECTIVES: I. Assess the efficacy of thioctic acid in treating a single patient with mitochondrial myopathy.
    Detailed Description
    PROTOCOL OUTLINE: The patient receives one thioctic acid tablet 3 times a day. If patient experiences no side effects after 1 week, 2 tablets are administered and given 3 times a day over 3 months for compassionate use. The patient is followed at weeks 3, 8, and 12.

    6. Conditions and Keywords

    Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
    Mitochondrial Myopathy
    Keywords
    inborn errors of metabolism, mitochondrial myopathy, rare disease

    7. Study Design

    Primary Purpose
    Treatment
    Study Phase
    Not Applicable
    Enrollment
    1 (false)

    8. Arms, Groups, and Interventions

    Intervention Type
    Drug
    Intervention Name(s)
    thioctic acid

    10. Eligibility

    Sex
    Male
    Minimum Age & Unit of Time
    0 Years
    Accepts Healthy Volunteers
    No
    Eligibility Criteria
    PROTOCOL ENTRY CRITERIA: --Disease Characteristics-- Long standing ophthalmoparesis and fatiguable weakness of the limbs Mild ataxia and no central nervous system involvement History of mitochondrial DNA deletion and a measurable biochemical defect of the respiratory chain Steady deterioration in skeletal muscle mass and power over 5 years --Prior/Concurrent Therapy-- Previous participation in studies of muscle disease natural history (CRC Protocol 183A)
    Overall Study Officials:
    First Name & Middle Initial & Last Name & Degree
    Robert Griggs
    Organizational Affiliation
    University of Rochester
    Official's Role
    Study Chair

    12. IPD Sharing Statement

    Learn more about this trial

    Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy

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